Public & philanthropic financial contribution to the development of new drugs 1: Methodology & 3 Case Studies

Research area: HTA-methods & steering instruments

Duration: February 2019 - May 2019
Language: English
Publications:
LBI-HTA Project report No. 120: https://eprints.aihta.at/1214/

Schmidt L, Wild C (2020): Assessing the public and philanthropic financial contribution to the development of new drugs: a bibliographic analysis. Science, Technology & Public Policy (STPP), 4 (1): 8-14.

Background:
Until a few years ago, the discussion on "access to medicines" was associated with the access to cheaper (generic) vital medicines (HIV, tuberculosis, etc.) in developing countries. For some time now Western countries and institutions (OECD (1), European Parliament (2,3), US institutions (4)) have also been dealing with access to medicines in numerous public discussions, events and reports. To react on the "non-sustainable" (= intolerable) drug prices with few actual innovations, in 2017, a committee of EC experts dealt with the internationally discussed solutions and their potential effects (5). The role of research and development (R & D) is given particular attention in the EC report: R & D spending is mostly used as a justification to justify the high drug prices. The real - resource-consuming and high-risk - basic research takes place predominantly in the public sector (in universities and corresponding publicly funded research institutions). It is well known that basic research is publicly funded to a large extent. Exact numbers were not yet available (6).

Project objectives: The project objective is to collect data and figures on public contributions to drug discovery and development.

Methods:

Phase 1: Development of methods (search strategy and sources) in which the contribution of public research funding of new NMEs can be determined

Phase 2: Piloting of the analysis approach developed in phase 1 for a selection of new active substances given an approval by the European Medicines Agency (EMA). Selection of drugs (NMEs only): around 3 (paediatric) orphan drugs approved between 2013-2017 (concrete products to be determined in detail after Phase 1).

Result: Short report of methods and results, in form of a publication

Milestones:

• Phase 1: August to mid September 2018
• Phase 2: Mid September to end November 2018

References:

1. Organisation for Economic Co-operation and Development (OECD), Sustainable access to innovative therapies (under public consultation). 2018.
2. European Parliament, Links between Pharmaceutical R&D Models and Access to Affordable Medicines, Study for the ENVI Committee. In Directorate-General for Internal Policies: Policy Deparment - Economic and Scientific Policy (ed). 2016.
3. European Parliament, Report on EU options for improving access to medicines (2016/2057(INI)). In Report of the ENVI Committee (ed). 2017.
4. National Academy of Sciences, Making Medicines Affordable, A National Imperative (2018).  National Academy Press (NAP) (ed). Washington DC: 2018.
5. Expert Panel on Effective Ways of Investing in Health (EXPH), Innovative Payment Models for High-Cost Innovative Medicines, European Commission (EC) (ed). Brussels: 2018.
6.  Cleary E, Beierlein J, Khanuja N, et al. Contribution of NIH funding to new drug approvals 2010–2016. Proceedings of the National Academy of Sciences (PNAS) 2018; February 12.